Cambridge Healthtech Institute’s 8th Annual

Late Stage Research Strategy and Operations

RWE for Regulatory Decisions, Market Access and Pharmacovigilance

February 19-20, 2019

Real world evidence solutions have changed the design and execution of peri-approval and post-marketing research. Real time real world data generation, pragmatic trials, and next generation databases open new opportunities for post-marketing research as well as regulatory and market access needs. Data generated in peri-approval real world data-based studies is essential for multiple stakeholders within and outside pharmaceutical companies, such as regulatory agencies, payers, health care management organizations, formulary inclusion decision makers, healthcare professionals, and patients. Cambridge Healthtech Institute’s 8th Annual Late Stage Research Strategy and Operations conference is designed to facilitate knowledge exchange around all aspects of peri-approval studies and major application of their outcomes/generated data.

Final Agenda

Stay on and attend Part 2 (Wed-Thurs): Leveraging RWD for Clinical and Observational Research

Scientific Advisory Board

Cathy Critchlow, PhD, Vice President, Center for Observational Research, Amgen  

Hui Cao, PhD, Executive Director, Real World Evidence, Global Medical Affairs, Novartis Pharmaceuticals  

Charles Makin, Global Head, Real World Evidence Strategy, Biogen  

Marc Berger, MD, Chair, Real World Evidence Advisory Committee, SHYFT Analytics  

Monday, February 18

9:00 am 7:15 pm Registration Open

2:005:00 pm User Group Meetings

Shared Investigator Platform User Forum

Trifecta Annual User Group Forum

5:006:15 pm Pre-Conference Plenary Keynote Panel & Participant Engagement Award

6:157:15 pm SCOPE’s Kick-off Networking Happy Hour Hosted by CHI (Sponsorship Opportunities Available)

7:15 pm Close of Day

Tuesday, February 19

7:15 am Registration Open and Morning Coffee

8:15 Opening Plenary Keynotes

9:35 Grand Opening Coffee Break in the Exhibit Hall


10:35 Chairperson’s Remarks

Charles Makin, Global Head, Real World Evidence Strategy, Biogen

10:40 RWD: Studies for Formulary Decision Making and Health Technology Assessment – A US Perspective

Marciniak_MartinMartin Marciniak, PhD, Vice President, US Medical Affairs, Customer Engagement, Value, Evidence & Outcomes, GSK

HTA is heavily dependent on real world data (RWD) in part because of its focus on measuring outcomes that extend past that offered by traditional clinical trial data or systematic reviews. RWD, in its many forms (e.g., pragmatic, observational, social media, other) is increasingly viewed as an important part of this process, particularly outside of the United States. What does this mean for the US, and its nascent HTA infrastructure?

11:10 Considerations for Use of Real World Evidence to Inform Regulatory Decisions

Critchlow_CathyCathy Critchlow, PhD, Vice President, Center for Observational Research, Amgen

Real world evidence (RWE) will be increasingly used in regulatory submissions seeking approval for a new indication, label expansion, or new product marketing authorization. The 21st Century Cures Act places additional focus on the use of RWE in regulatory decision making. While growing availability of data and sophistication of analytic tools have transformed RWE generation, challenges impeding full realization of benefit from RWE involve issues such as data quality and what constitutes “substantial evidence” when using RWE as supporting or pivotal evidence. Considerations to help ensure the advancement of suitable RWE use cases to enable appropriate impact of RWE in regulatory decision making will be discussed.

11:40 Use of Real World Data (RWD) to Assess Cardiovascular Safety of Prucalopride to Support a New Drug Application in the US

Spalding_WilliamWilliam Spalding, MS, Director, Outcomes Research-Epidemiology, Shire

Prucalopride belongs to the 5HT4 agonist drug class, and 2 previously approved 5HT4 agonist drugs have been removed from the market for increased risk of adverse cardiovascular (CV) events. Shire, in agreement with the FDA, sponsored a CV safety study based on RWD from clinical use of prucalopride in European markets where prucalopride has been marketed since 2009. This talk discusses rationale for use of RWD to assess drug safety.

12:10 pm Using Technology to Maximize Patient Choice While Minimizing Burden in Post-Approval Research

Watson_ChrisChris Watson, PhD, Director, Product Strategy, Digital Patient Product Management, ERT

With the rise in importance of prospective patient data capture as part of real-world evidence analysis how can we make it simpler and easier for patients to participate - and remain engaged - in post-approval research? What role does technology play and how can advances in consumer technology simplify the process for patients, investigators and sponsors?

12:40 Transition to Lunch

12:45 LUNCHEON PRESENTATION: Optimizing Real World Research Design: Methodologic and Regulatory Perspectives

Stuart McCully, PhD, Vice President, Regulatory Advisory Services, Syneos Health

David Thompson, PhD, Senior Vice President, Real World Evidence Advisory, Syneos Health

1:25 Coffee and Dessert Break in the Exhibit Hall


2:05 Chairperson’s Remarks

Hui Cao, PhD, Executive Director, Real World Evidence, Global Medical Affairs, Novartis Pharmaceuticals

2:10 A Practical Paradigm of Using RWE for Seeking Regulatory Decisions in Label Expansion or New Indication

Cao_HuiHui Cao, PhD, Executive Director, Real World Evidence, Global Medical Affairs, Novartis Pharmaceuticals

21st Century Cure Act required the FDA to provide a framework and guidance for evaluating RWE in the context of drug regulation to support approvals of new indications for previously approved drugs and to support or fulfill post-approval study requirements. We established a practical paradigm to guide drug projects teams in developing high-quality RWE proposals to the FDA for these two areas.

2:40 Methodologies of Incorporating Patient-Focused Drug Development into Regulatory Submissions

Freeman_EmilyEmily Freeman, PhD, Director, Patient Centered Outcomes, HEOR, Abbvie

With the approval of PDUFA VI and the passage of the 21st Century Cures Act, the FDA is mandated to include the patient voice in its consideration of new medical product development.

3:10 Real World Evidence and the FDA: Where Are We Today?

Martin_DavidDavid Martin, MD, Associate Director, Real World Evidence Analytics, Office of Medical Policy, US FDA Center for Drug Evaluation & Research

This presentation will provide an overview of the framework for the FDA evaluation of RWD and RWE mandated by the 21st Century Cures Act. Early lessons from FDA demonstration projects and review activities will also be described.

3:40 Presentation to be Announced


4:10 Find Your Table and Meet Your Moderator

4:15 Interactive Breakout Discussion Groups

Concurrent breakout discussion groups are interactive, guided discussions hosted by a facilitator or set of co-facilitators to discuss key issues presented earlier in the day’s sessions. Delegates will join a table of interest and become an active part of the discussion. Bring your pharma, biotech, CRO, site, hospital or patient perspective to each of the discussions. To get the most out of this interactive session and format, please come prepared to share examples from your work, vet some ideas with your peers, be a part of group interrogation and problem solving, and most importantly, participate in active idea sharing.

5:00 Welcome Reception in the Exhibit Hall

6:30 Close of Day

Wednesday, February 20

7:15 am Registration Open

7:45 BREAKFAST PRESENTATION: ICF Authoring - The Next Evolution of Informed Consent

Delente_EricEric Delente, President, Patient Consent, DrugDev, An IQVIA Company

This session will explore how the recent introduction of informed consent form (ICF) authoring systems, including IQVIA ICF Author, have evolved the informed consent process by allowing sponsors and CROs to create, distribute, and track ICFs from a centralized portal, whether using paper forms or eConsent. It will also explore the features and benefits of these portals, showing why sponsors and CROs should consider adoption.

8:15 Session Break


8:20 Chairperson’s Remarks

Chairperson to be Announced

8:25 Real World Considerations for Using Real World Common Data Models and Rapid Analytics

Kahler_KristijanKristijan Kahler, PhD, Head, Outcomes Evidence & Analytics, RWE & Digital, Global Medical Affairs, Novartis

Use of real world data (RWD) has evolved over the last several decades to a point where the body of evidence for any research question can easily be addressed across multiple databases with the use common data models (CDMs). These CDMs can enable rapid insight generation by improving interoperability across different data types and different geographies, but also come with some important practical limitations.

8:55 Using Data and Analytics to Synthesize Real-World Evidence

Gamerman_VictoriaVictoria Gamerman, Associate Director, Biostatistics, Head, Health Informatics & Analytics, Boehringer-Ingelheim Pharmaceuticals, Inc.

In this presentation, learn about an integrated approach to understanding real-world evidence needs throughout the medical organization. Two case studies will be highlighted: (1) an observational US-based registry in a rare disease to capture the real-world patient journey; and (2) a global survey of patients and physicians in a chronic condition. A future outlook on RWD standards and the role of Big Data in RWE generation will be shared.

9:25 Identifying Breast Cancer Stage and Biomarker Status in Administrative Claims Data Using Predictive Modeling

de_Luise_CynthiaCynthia de Luise, PhD, Senior Director, Epidemiology, Worldwide Safety and Regulatory, Pfizer, Inc.

Claims databases lack stage and receptor status to identify cancer populations. From Anthem’s Cancer Care Quality program validation sample, we used regression and machine learning techniques to construct a predictive model to identify advanced stage ER+/HER2- breast cancer (ASBC) patients. The model (c-statistic=0.93; PPV=91%; Sensitivity=54%) was applied to the HealthCore Integrated Research Database (HIRD) to identify an ASBC cohort. Predictive modeling can identify cancer stage and receptor status in claims.

9:55 Presentation to be Announced

10:25 Coffee Break in the Exhibit Hall


11:20 Chairperson’s Remarks

Sean Zhao, PhD, Head, US Patient Safety Surveillance, US Medical Affairs, AstraZeneca Pharmaceuticals, Inc.

11:25 Considerations and Challenges Associated with the Implementation of an Integrated Value-Based Pharmaceutical Data and Evidence Strategy

Hebden_TonyTony Hebden, PhD, Vice President, Health Economics & Outcomes Research, AbbVie

Pharmaceutical companies have identified the need to define value to key access stakeholders as critical to ensuring appropriate patient access to new medications. This approach requires that companies not only define value in terms of the classical RCT evidence required to obtain regulatory approval, but also develop new processes, infrastructure and governance to ensure less traditional types of evidence are generated and leveraged appropriately.

11:55 Cross-Company Coordination of Post Marketing Research Efforts

Zhao_SeanSean Zhao, PhD, Head, US Patient Safety Surveillance, US Medical Affairs, AstraZeneca Pharmaceuticals, Inc.

There are many types of post marketing research activities, including clinical trials, patient registries, epidemiology studies, health economic and outcome researches, etc., having been used for multiple purposes, i.e., fulfilling regulatory post marketing requirements (PMRs), supporting in risk management activities, closing clinical evidence gaps, generating real life clinical evidences in supporting formulary decisions, etc. It is important to strategically plan and implement these studies and activities through a cross-company coordination. The presentation will discuss how to involve cross-company functions in post marketing research activities and when a post marketing research activity’s scientific purposes can be combined with commercial goals and when they should be separated.

12:25 pm Transition to Lunch


Quigley_Jane_PH4Jane Quigley, Senior Vice President, Medical Informatics, PRA Health Sciences

1:10 Coffee and Dessert Break in the Exhibit Hall

2:10 Close of Conference

Stay on and attend Part 2 (Wed-Thurs): Leveraging RWD for Clinical and Observational Research


“SCOPE Featured Author”
Emmanuel Fombu, MD,
Director, Digital Health Solutions

Signature Sponsors



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Mendel Health

PRA Health Sciences 

Syneos Health